molecules of the month

What is it? Saridegib (IPI-926, patidegib) is a Smoothened receptor (SMO) antagonist and hedgehog pathway inhibitor currently in Ph. III trials for Gorlin syndrome. The molecule is a semisynthetic analog of the naturally-occuring SMO antagonist, cyclopamine, and has a long history and originates from Infinity Pharmaceuticals. After a clinical disappointment as an oral therapy for myelofibrosis and chondrosarcoma in 2012, Infinity out-licensed the molecule to PellePharm in 2013, a company later backed by BridgeBio. Recently, it again changed hands from BridgeBio/PellePharm to Sol-Gel Technologies in a ~$75M transaction. Sol-Gel plans to continue development of the molecule for Gorlin syndrome and basal cell carcinomas as a topical treatment to avoid systemic toxicities, giving this older molecule hope of a new life.…